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Webinar: How to achieve high-throughput single-cell sequencing

Drug Discovery World

It will also feature an introduction by Melanie Masuda, Director of Market Development, ScaleBio. Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel.

Genome 52
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CRISPR screening: Achieving high-throughput single-cell sequencing

Drug Discovery World

It will also feature an introduction by Melanie Masuda, Director of Market Development, ScaleBio. Pooled CRISPR screens offer the ability to knock out or perturb many genes in a single experiment from several hundred to tens of thousands of targets in parallel.

Genome 52
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CAMP4’s funding influx paves the way for tapping regulatory RNA to treat urea cycle disorders

Pharmaceutical Technology

The most common marketed drugs in this space aim to address the hyperammonaemia caused by the defective genes in this disorder. Drugs for dialysis, amino acid supplements, and drugs which convert blood ammonia are marketed right now as the go-to urea cycle disorder treatments.

RNA 245
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Turning science into business: Amplifying mRNA by targeting regRNAs

Drug Discovery World

DDW’s Diana Spencer speaks to Josh Mandel-Brehm , CEO of Massachusetts-based biotech CAMP4, to understand the role regulatory RNAs play in gene expression and how the founders overcame the challenge of launching a business based on brand new science. DS: How easy was it to transfer theoretical research into a viable business?

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Alnylam identifies gene to lower abdominal fat and decrease cardiovascular risk

Pharmaceutical Technology

Alnylam Pharmaceuticals and collaborators have identified rare mutations in the INHBE gene that is expressed in the liver, related to a lower waist-to-hip ratio for body mass index (BMI), which is often used as an indicator of abdominal fat and is correlated with the risk of type 2 diabetes (T2D) and coronary heart disease.

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Gene editing: beyond the hype

pharmaphorum

Genome editing is an exciting but still nascent field, and companies in the area face as many obstacles as they do opportunities. Then over the next two or three years, gene therapy was accepted as something that companies got involved in, and several biotechs have been bought up by big pharma.”. The genomic medicine journey.

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Right on target: The shift to precision pharmaceuticals

Drug Discovery World

Think how cell and gene therapies or monoclonal antibodies have become targeted medicines for cancer and you’ll get an idea of the shifting pharmaceutical market. Its convergence with genomics makes sense and we have now multiple examples of AI being used to inform prescribing, diagnostics and drug discovery/ development.